RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
    1.
    发明授权
    RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 有权
    使用短干扰核酸(siNA)的RNA干扰介导的丙型肝炎病毒(HCV)表达的抑制

    公开(公告)号:US07935812B2

    公开(公告)日:2011-05-03

    申请号:US12418477

    申请日:2009-04-03

    IPC分类号: C07H21/04 C07H21/02 A61K48/00

    摘要: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of hepatitis C virus (HCV) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in hepatitis C virus (HCV) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.

    摘要翻译: 本发明涉及用于研究,诊断和治疗对丙型肝炎病毒(HCV)基因表达和/或活性的调节作用的性状,疾病和病症的化合物,组合物和方法。 本发明还涉及与涉及丙型肝炎病毒(HCV)基因表达途径或介导的其他细胞过程的基因的表达和/或活性的调节作用的性状,疾病和病症有关的化合物,组合物和方法 维持或发展这些性状,疾病和状况。 具体地,本发明涉及能够介导或介导针对丙型肝炎病毒(HCV)基因表达的RNA干扰(RNAi)的双链核酸分子,包括这种小核酸分子的混合物和这种小的核酸分子的脂质纳米颗粒(LNP)制剂 核酸分子。

    RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA)
    2.
    发明申请
    RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA) 审中-公开
    使用短干扰核酸(siNA)介导的流感病毒基因表达抑制的RNA干扰

    公开(公告)号:US20070099858A1

    公开(公告)日:2007-05-03

    申请号:US11242567

    申请日:2005-10-03

    IPC分类号: A61K48/00 C07H21/02

    摘要: This invention relates to compounds, compositions, and methods useful for modulating influenza virus gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of influenza virus genes, such as viral and host genes associated with the maintenance or development of influenza virus infection and related diseases and conditions in a subject or organism.

    摘要翻译: 本发明涉及可用于使用短干扰核酸(siNA)分子调节流感病毒基因表达的化合物,组合物和方法。 特别地,本发明的特征在于小核酸分子,例如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)和短发夹RNA(shRNA) )分子和用于调节流感病毒基因表达的方法,例如与维持或发展流感病毒感染以及受试者或生物体中的相关疾病和病症相关的病毒和宿主基因。

    RNA interference mediated inhibition of histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA)
    4.
    发明申请
    RNA interference mediated inhibition of histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA) 审中-公开
    RNA干扰介导使用短干扰核酸(siNA)抑制组蛋白脱乙酰酶(HDAC)基因表达

    公开(公告)号:US20060148743A1

    公开(公告)日:2006-07-06

    申请号:US11217936

    申请日:2005-09-01

    IPC分类号: A61K48/00 C07H21/02

    摘要: This invention relates to compounds, compositions, and methods useful for modulating histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA) molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HDAC genes, such as HDAC genes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism such as cancer, proliferative disease, and age related disease.

    摘要翻译: 本发明涉及可用于使用短干扰核酸(siNA)分子调节组蛋白脱乙酰酶(HDAC)基因表达的化合物,组合物和方法。 特别地,本发明的特征在于小核酸分子,例如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微RNA(miRNA)和短发夹RNA(shRNA) )分子和方法用于调节HDAC基因的表达,例如与受试者或生物如癌症,增殖性疾病和年龄相关疾病中的疾病,病症,性状和病症的维持或发展相关的HDAC基因。

    RNA Interference Mediated Inhibition Of Gene Expression Using Multifunctional Short Interfering Nucleic Acid (Multifunctional siNA)
    9.
    发明申请
    RNA Interference Mediated Inhibition Of Gene Expression Using Multifunctional Short Interfering Nucleic Acid (Multifunctional siNA) 审中-公开
    RNA干扰介导的使用多功能短干扰核酸(多功能siNA)的基因表达抑制

    公开(公告)号:US20110118335A1

    公开(公告)日:2011-05-19

    申请号:US12947609

    申请日:2010-11-16

    摘要: The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to multifunctional short interfering nucleic acid (multifunctional siNA) molecules that modulate the expression of one or more genes in a biologic system, such as a cell, tissue, or organism via RNA interference (RNAi). The bifunctional short interfering nucleic acid (multifunctional siNA) molecules of the invention can target more than one regions of nucleic acid sequence in a single target nucleic acid molecule or can target regions of nucleic acid sequence in differing target nucleic acid molecules. The self multifunctional siNA molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

    摘要翻译: 本发明涉及可用于调节各种应用中的基因表达的方法和基于核酸的试剂,包括在治疗,兽医,农业,诊断,靶标验证和基因组发现应用中的用途。 具体地,本发明涉及通过RNA干扰(RNAi)调节生物系统例如细胞,组织或生物体中的一种或多种基因的表达的多功能短干扰核酸(多功能siNA)分子。 本发明的双功能短干扰核酸(多功能siNA)分子可以靶向单个靶核酸分子中的多于一个核酸序列区域,或者靶向不同靶核酸分子中核酸序列的区域。 自身多功能siNA分子可用于治疗对细胞,组织或生物中基因表达或活性的调节作出反应的任何疾病或病症。

    COMPOSITIONS AND METHODS FOR POTENTIATED ACTIVITY OF BIOLOGICALLY ACTIVE MOLECULES

    公开(公告)号:US20100015218A1

    公开(公告)日:2010-01-21

    申请号:US12526869

    申请日:2008-02-15

    CPC分类号: C12N15/88

    摘要: The present invention relates to novel compositions and methods for potentiating the activity of biologically active molecules in conjunction with one or more delivery vehicles and one or more carrier molecules. Specifically, the invention features the use of a carrier molecule in combination with a delivery vehicle and a biologically active molecule of interest to potentiate the activity of the biologically active molecule. The carrier molecule can be biologically inert, inactive, or attenuated; or can alternately be biologically active in the same or different manner than the biologically active molecule of interest. Specifically, the invention features novel particle forming delivery agents including cationic lipids, microparticles, and nanoparticles that are useful for delivering various biologically active molecules to cells in conjunction with a carrier molecule. The invention also features compositions, and methods of use for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity in a subject or organism that are delivered intracellularly in conjunction with a carrier molecule. In various embodiments, the invention relates to novel cationic lipids, microparticles, nanoparticles and transfection agents that effectively transfect or deliver biologically active molecules, such as antibodies (e.g., monoclonal, chimeric, humanized etc.), cholesterol, hormones, antivirals, peptides, proteins, chemotherapeutics, small molecules, vitamins, co-factors, nucleosides, nucleotides, oligonucleotides, enzymatic nucleic acids, antisense nucleic acids, triplex forming oligonucleotides, 2,5-A chimeras, allozymes, aptamers, decoys and analogs thereof, and small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules, to relevant cells and/or tissues, such as in a subject or organism, in conjunction with one or more carrier molecules. Such novel cationic lipids, microparticles, nanoparticles and transfection agents that are used in conjunction with one or more carrier molecules are useful, for example, in providing compositions to prevent, inhibit, or treat diseases, conditions, or traits in a cell, subject or organism.